There is a general lack of therapies for optic neuropathies. Glaucoma is treated in part by lowering intraocular pressure. Optic neuritis is managed by corticosteroids, but this does not affect the long-term course of the disease. Compressive optic neuropathy is treated by removing the tumor or aneurysm pressing on the optic nerve or chiasm. All other optic neuropathies, including nonarteritic anterior ischemic optic neuropathy (NAION), represent unmet medical needs. (Levin La. Axonal loss and neuroprotection in optic neuropathies. Can J Ophthalmol. 2007, 42(3):403-8).
PCT Publication Nos. WO 2008/050329 and WO 2009/044392 are directed to inhibitors of pro-apoptotic genes and disclose double-stranded RNA molecules targeting, inter alia, Caspase 2.
PCT Publication No. WO 2010/048352 is directed to compositions and methods of treating ocular diseases and discloses, inter alia, the chemically modified, double-stranded RNA compound QPI-1007 targeting the Caspase 2 gene.
Ahmed Z. et al., (Cell Death and Disease (2011) 2, e173) suggest that retinal ganglion cell (RGC) apoptosis induced by optic nerve injury in a rat model of optic nerve transection, involves activation of Caspase 2, and that synthetic double stranded RNA compounds designed to inhibit expression of Caspase 2 represent potential neuroprotective agents for intervention in human diseases involving RGC loss.